Hemophilia B is a rare bleeding disorder that causes blood to clot more slowly than usual. If you have this condition, you may have bleeding after an injury or surgery. You may have bleeding after a minor injury or even without an injury.

Hemophilia B is caused by genetic mutations in the F9 gene. This gene gives your body instructions for producing factor IX. This is one of multiple clotting factors that help your blood to clot. If you have hemophilia B, your body does not produce enough factor IX.

Factor IX replacement therapy is the first line of treatment. Your doctor will give you high amounts of factor IX concentrate to stop bleeding. They may prescribe prophylactic therapy, which involves taking regular doses of factor IX concentrate to help prevent bleeds.

If you need multiple prophylactic injections per week, factor IX therapy is inconvenient. Some people with the blood disorder have neutralizing antibodies that block the action of factor IX. Factor IX therapy is less effective because of these.

New factor IX products and other therapies have been developed to improve treatment options.

The latest research findings and novel treatments for this condition are available.

The earliest factor IX products were derived from human blood.

Scientists developed a product called rFIX, which is genetically engineered.

The FDA approved the first rFIX product in the 1990s.

Scientists have modified rFIX to create products with longer half-life. rFIX products have a standard half-life. You need less frequent doses.

The FDA has approved multiple rFIX products.

  • eftrenonacog alfa (Alprolix; rFIX-Fc), approved in 2014
  • albutrepenonacog alfa (Idelvion; rFIX-FP), approved in 2016
  • nonacog beta pegol (Rebinyn; N9-GP), approved in 2017

The products are infused into a vein.

Researchers are continuing to study the long-term effects of these products, according to a 2021 review.

Scientists are testing other rFIX products with an extended half-life. This product is injected into your skin.

A small phase 1/2a clinical trial found that daily injections of DalcA were safe and effective for increasing factor IX in people with hemophilia B. More research is needed to evaluate this treatment.

Factor IX is one of the factors that helps clot your blood. Treatments for Hempophilic B increase the availability of other clotting factors to help stop bleeding.

The factor VIIa is included. If you have factor IX inhibitors, your doctor may prescribe rFVIIa to treat and prevent bleeding episodes.

The FDA approved two rFVIIa products.

  • NovoSeven RT, approved in 1999
  • eptacog beta (SEVENFACT), approved in 2020

SevenFACT is only approved for adults and adolescents, but recent research suggests it may be safe for younger people.

In a phase 3 clinical trial, 25 children with hemophilia A or B under age 12 received SEVENFACT to treat a mild to moderate bleeding episode. The treatment was successful within 24 hours for at least 97% of children who received it. No participants experienced serious side effects.

rFVIIa products are being developed that will last longer in your body than NonoSevenRT or SevenFACT. The products include MOD-5014 and marzeptacog alfa.

There is ongoing research on these products.

Factor Xa and factor IXa are both blocked by antithrombin.

Fitusiran lowers levels of antithrombin. This helps with blood clotting and limits bleeding.

In 2022, researchers reported the results of two phase 3 clinical trials on fitusiran at a conference of the International Society on Thrombosis and Haemostasis. They found that monthly doses of fitusiran were linked to fewer bleeding episodes in people with hemophilia A or B. Those who took fitusirin also required fewer doses of clotting factor to treat bleeds.

fitusirin works in people with factor IX inhibitors as well as those without.

Fitusirin is not approved by the FDA for treating hemophilia A or B.

Researchers are studying it to learn more about its safety and efficacy.

The tissue factor pathway is a factor that limits the action of the clotting factor Xa.

Concizumab blocks the TFPI. Factor IX can cause blood to clot, but blocking the TFPI will help.

Phase 1 and 2 clinical trials found that concizumab was effective for reducing bleeds in people with hemophilia A or B, report the authors of a 2022 review.

People with factor IX inhibitors may be able to get Concuzimab.

There are ongoing Phase 3 clinical trials.

Researchers are studying other anti-TFPI antibodies.

  • Befovacimab is a drug.
  • marstacimab is a drug.
  • The person is named MG1113

There is more research needed to evaluate the safety and efficacy of these medications.

Gene therapy corrects genetic defects that cause diseases.

“A doctor uses a modified virus to deliver genes into your body’s cells. They may remove or inactivate faulty genes.”

Successful gene therapy for hemophilia B could provide long-term or potentially permanent benefits by replacing your faulty F9 gene with a nonfaulty gene. This would allow your body to produce enough factor IX on its own, with fewer or no infusions of factor IX.

There are multiple approaches for treating Hemophilia B.

For example, researchers found gene therapy increased circulating factor IX levels to 1 to 6% of the normal level for at least 3 years. People who received the highest dose of the therapy had more than 90% fewer bleeding episodes. They also required fewer treatments with factor IX concentrates.

Gene therapy is not currently available to treat a bleeding disorder.

There is more research that needs to be done to study its long-term safety.

It is important to get treatment for the bleeding disorders like hemophilia B to prevent and manage bleeding episodes, lower your risk of complications, and improve your quality of life.

Factor IX replacement and prophylactic therapy are the main treatments. Some people make treatments less effective.

New and experimental therapies may help improve treatment options.

Talk to your doctor about treatment options. They might encourage you to take part in a clinical trial to receive an experimental treatment that has not been approved.

Your doctor can help you learn more about the risks and benefits of different treatments for the bleeding disorders.